528 millions of euros invested since 1990

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We've funded more than 2500 projects and 1600 researchers to give people affected by a rare genetic condition a new hope.

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A new hope for Isaac

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When Isaac was born, his parents Andrew and Ellen we're worried that their baby was growing too fast. But now they have a new hope.

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What we do

Our mission

We were made to give people affected by a rare genetic condition an answer – treatment for their condition. Rare genetic conditions are not statistically relevant when considered individually, but the same can’t be said about a person’s life.

Projects funded

We support the best projects chosen using a rigorous and transparent selection process, following consultation with an internationally-renowned committee of qualified scientists.

Institutes

We have founded two institutes that are global centres of excellence in the study of rare genetic conditions: The San Raffaele Telethon Institute For Gene Therapy in Milan and Telethon Institute of Genetics and Medicine in Pozzuoli (NA).

Tommaso and his return to Normality

Patients

Tommaso was treated for ADA-SCID in February 2016 at the San Raffaele Telethon Institute in Milan (SR – Tiget).

News

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15.02.19

Tigem and SR-Tiget involved in UPGRADE project, funded by the European Union

Fondazione Telethon announces the launch of the UPGRADE project, funded by the European Union with 15 million euros, aiming at developing more precise and effective gene therapies approaches.

From Telethon Foundation

21.01.19

Beta Thalassemia: clinical trial provides encouraging evidence for efficacy of gene therapy

Gene therapy, especially if administered early, could be an effective treatment strategy for beta thalassemia, a genetic disease that is quite common in Mediterranean countries, affecting over 7000 patients in Italy only.

From Research

14.11.18

A new ingredient to potentiate gene therapy in stem cells

A new study published by the research team led by Anna Kajaste-Rudnitski at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, shows that a naturally occurring compound significantly increases the efficiency of lentiviral vector-mediated gene transfer in blood stem cells.